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BioZ - study
Biologics during pregnancy
Immune profiling of peripheral and endometrial immune cells of women with psoriasis treated with biologics

To identify the effect of psoriasis and the treatment with biologics on the pre-pregnancy uterine immune environment of psoriasis patients.

An Observational Study in Participants with Ryanodine Receptor 1-Related Myopathies (RYR1-RM) to Determine Optimal Endpoint Measurements.

Primary objective* To determine the extent to which muscle strength is affected in patients with RYR1-RM with autosomal dominantmutations and describe measurements over time.Secondary objectives* To determine the extent to which fatigue and…

A-mode ultrasound measurement of jugular venous pulse wave versus invasive hemodynamics: a comparison between pulmonary hypertension and healthy controls, and hemodynamic modulation (LUMINA-study)

This study aims to evaluate A-mode ultrasound for assessing JVPW characteristics as a non-invasive tool to discriminate between healthy subjects and those with pulmonary arterial hypertension patients, and to assess if JVPW changes relate to…

UNIFAI

To generate a large, international Friedreich*s ataxia patient database (including healthy controls), alongside an integrated clinical and natural history database, which is linked to a biological samples and imaging repository, hereby creating a…

A Multicenter, Prospective, Longitudinal, Observational Study in Children and Adults with Stargardt Disease Secondary to Mutation in the ABCA4 Gene

Primary Objectives• To evaluate prognostic factors of disease progression in subjects with Stargardt disease secondary to mutation in the ABCA4 gene • To further characterize the STGD1 patient population for future interventional clinical studies